Chelsea completes successful NORTHERA pre-NDA assessment with FDA

Dec 20 2010

• Chelsea to File for US Marketing Approval of Northera in Symptomatic Neurogenic Orthostatic Hypotension in Q2 2011 Based on Efficacy Data from Phase III Studies 301 and 302
• FDA Will Not Require Study 306 as Second Pivotal Study for NDA
• Management to Host Conference Call at 9:00 AM ET

Chelsea Therapeutics International, Ltd. (Nasdaq:CHTP) announced it has completed a pre-NDA assessment with the U.S. Food and Drug Administration (FDA) and intends to accelerate its new drug application (NDA) for NORTHERA™(droxidopa) for the treatment of symptomatic, neurogenic orthostatic hypotension (NOH), a chronic and potentially debilitating drop in blood pressure upon standing commonly associated Parkinson's disease, multiple system atrophy and pure autonomic failure.  

At the meeting, the FDA agreed that the proposed NDA for Northera could be submitted based on combined data from Chelsea's two completed Phase III studies in NOH, Study 301 and Study 302, without the need for any further efficacy studies. During the meeting, the agency did request and Chelsea agreed to supply top-line results from a QTc study at the time of the 90-day Safety Update and conduct a post-marketing study to evaluate the clinical pharmacology of Northera in renally impaired patients.

"The successful outcome of our pre-NDA meeting with the FDA reflects the strength of the data already generated by our pivotal program and marks a significant step forward for Chelsea," commented Dr. Simon Pedder, president and CEO of Chelsea Therapeutics. "We believe that the Phase III trials we have already completed, combined with the extensive Japanese and European data available to us, clearly demonstrate Northera's meaningful clinical benefit to patients whose day to day lives are severely impacted by the signs and symptoms of neurogenic orthostatic hypotension. Furthermore, based on feedback received during the meeting and subsequent correspondence, we believe the safety profile of Northera is unlikely to result in a black box warning for supine hypertension in its label. We appreciate the guidance that the FDA has given the company as it prepares this new NDA, and look forward to working with the FDA as we seek marketing approval for Northera in the US in 2011."

Northera has previously been granted Orphan Drug Designation and has received Fast Track designation from the FDA. Fast Track designation is designed to facilitate the review of products that address serious or potentially life-threatening conditions for which there is an unmet medical need.

Study 301 was reviewed by the FDA and awarded a Special Protocol Assessment (SPA) in February 2008. The FDA subsequently confirmed that the SPA remained in effect following the protocol amendments and change in primary endpoint approved by the FDA in November 2009. An SPA provides a binding agreement that the study design, including trial size, clinical endpoints and/or data analyses is acceptable to support regulatory approval.